Using iPS Cells toward the Understanding of Parkinson’s Disease
Cellular reprogramming of somatic cells to human pluripotent stem cells (iPSC) represents an efficient tool for in vitro modeling of human brain diseases and provides an innovative opportunity in the identification of new therapeutic drugs. Patient-specific iPSC can be differentiated into disease-re...
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doaj-8bdf6564d8ed4b75b6cd1152ed44cf042020-11-25T00:52:31ZengMDPI AGJournal of Clinical Medicine2077-03832015-03-014454856610.3390/jcm4040548jcm4040548Using iPS Cells toward the Understanding of Parkinson’s DiseaseRoger Torrent0Francesca De Angelis Rigotti1Patrizia Dell'Era2Maurizio Memo3Angel Raya4Antonella Consiglio5Institute for Biomedicine of the University of Barcelona (IBUB), Barcelona Science Park, Barcelona 08028, SpainInstitute for Biomedicine of the University of Barcelona (IBUB), Barcelona Science Park, Barcelona 08028, SpainDepartment of Molecular and Translational Medicine, Fibroblast Reprogramming Unit, University of Brescia, Brescia 25123, ItalyDepartment of Molecular and Translational Medicine, Fibroblast Reprogramming Unit, University of Brescia, Brescia 25123, ItalyControl of Stem Cell Potency Group, Institute for Bioengineering of Catalonia (IBEC), Barcelona 08028, SpainInstitute for Biomedicine of the University of Barcelona (IBUB), Barcelona Science Park, Barcelona 08028, SpainCellular reprogramming of somatic cells to human pluripotent stem cells (iPSC) represents an efficient tool for in vitro modeling of human brain diseases and provides an innovative opportunity in the identification of new therapeutic drugs. Patient-specific iPSC can be differentiated into disease-relevant cell types, including neurons, carrying the genetic background of the donor and enabling de novo generation of human models of genetically complex disorders. Parkinson’s disease (PD) is the second most common age-related progressive neurodegenerative disease, which is mainly characterized by nigrostriatal dopaminergic (DA) neuron degeneration and synaptic dysfunction. Recently, the generation of disease-specific iPSC from patients suffering from PD has unveiled a recapitulation of disease-related cell phenotypes, such as abnormal α-synuclein accumulation and alterations in autophagy machinery. The use of patient-specific iPSC has a remarkable potential to uncover novel insights of the disease pathogenesis, which in turn will open new avenues for clinical intervention. This review explores the current Parkinson’s disease iPSC-based models highlighting their role in the discovery of new drugs, as well as discussing the most challenging limitations iPSC-models face today.http://www.mdpi.com/2077-0383/4/4/548induced pluripotent stem cellsParkinson’s diseaseLeucine-rich repeat kinase 2 (LRRK2)dopaminergic neurons |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Roger Torrent Francesca De Angelis Rigotti Patrizia Dell'Era Maurizio Memo Angel Raya Antonella Consiglio |
spellingShingle |
Roger Torrent Francesca De Angelis Rigotti Patrizia Dell'Era Maurizio Memo Angel Raya Antonella Consiglio Using iPS Cells toward the Understanding of Parkinson’s Disease Journal of Clinical Medicine induced pluripotent stem cells Parkinson’s disease Leucine-rich repeat kinase 2 (LRRK2) dopaminergic neurons |
author_facet |
Roger Torrent Francesca De Angelis Rigotti Patrizia Dell'Era Maurizio Memo Angel Raya Antonella Consiglio |
author_sort |
Roger Torrent |
title |
Using iPS Cells toward the Understanding of Parkinson’s Disease |
title_short |
Using iPS Cells toward the Understanding of Parkinson’s Disease |
title_full |
Using iPS Cells toward the Understanding of Parkinson’s Disease |
title_fullStr |
Using iPS Cells toward the Understanding of Parkinson’s Disease |
title_full_unstemmed |
Using iPS Cells toward the Understanding of Parkinson’s Disease |
title_sort |
using ips cells toward the understanding of parkinson’s disease |
publisher |
MDPI AG |
series |
Journal of Clinical Medicine |
issn |
2077-0383 |
publishDate |
2015-03-01 |
description |
Cellular reprogramming of somatic cells to human pluripotent stem cells (iPSC) represents an efficient tool for in vitro modeling of human brain diseases and provides an innovative opportunity in the identification of new therapeutic drugs. Patient-specific iPSC can be differentiated into disease-relevant cell types, including neurons, carrying the genetic background of the donor and enabling de novo generation of human models of genetically complex disorders. Parkinson’s disease (PD) is the second most common age-related progressive neurodegenerative disease, which is mainly characterized by nigrostriatal dopaminergic (DA) neuron degeneration and synaptic dysfunction. Recently, the generation of disease-specific iPSC from patients suffering from PD has unveiled a recapitulation of disease-related cell phenotypes, such as abnormal α-synuclein accumulation and alterations in autophagy machinery. The use of patient-specific iPSC has a remarkable potential to uncover novel insights of the disease pathogenesis, which in turn will open new avenues for clinical intervention. This review explores the current Parkinson’s disease iPSC-based models highlighting their role in the discovery of new drugs, as well as discussing the most challenging limitations iPSC-models face today. |
topic |
induced pluripotent stem cells Parkinson’s disease Leucine-rich repeat kinase 2 (LRRK2) dopaminergic neurons |
url |
http://www.mdpi.com/2077-0383/4/4/548 |
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